Androgen use in bone marrow failures and myeloid neoplasms: Mechanisms of action and a systematic review of clinical data

IF 6.9 2区 医学 Q1 HEMATOLOGY Blood Reviews Pub Date : 2023-11-01 DOI:10.1016/j.blre.2023.101132
Alessandro Bosi , Wilma Barcellini , Francesco Passamonti , Bruno Fattizzo
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Abstract

Despite recent advancements, treatment of cytopenia due to bone marrow failures (BMF) and myeloid neoplasms remains challenging. Androgens promote renewal and maturation of blood cells and may be beneficial in these forms. Here we report a systematic review of androgens use as single agent in hematologic conditions. Forty-six studies, mainly retrospective with various androgen types and doses, were included: 12 on acquired aplastic anemia (AA), 11 on inherited BMF, 17 on myelodysplastic syndromes (MDS), and 7 on myelofibrosis. Responses ranged from 50 to 70% in inherited BMF, 40–50% in acquired AA and MDS, while very limited evidence emerged for myelofibrosis. In acquired AA, response was associated with presence of non-severe disease; in MDS androgens were more effective on thrombocytopenia or mild to moderate anemia, whilst limited benefit was observed for transfusion dependent anemia. Toxicity profile mainly consisted of virilization and liver enzyme elevation, whilst the risk of leukemic evolution remains controversial.

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雄激素在骨髓衰竭和髓系肿瘤中的应用:作用机制和临床数据的系统回顾
尽管最近取得了进展,但由于骨髓衰竭(BMF)和髓系肿瘤引起的细胞减少症的治疗仍然具有挑战性。雄激素促进血细胞的更新和成熟,在这些形式中可能是有益的。在这里,我们报告了雄激素作为单一药物在血液学条件下使用的系统综述。纳入46项研究,主要是回顾性研究,涉及各种雄激素类型和剂量:12项关于获得性再生障碍性贫血(AA), 11项关于遗传性BMF, 17项关于骨髓增生异常综合征(MDS), 7项关于骨髓纤维化。遗传性BMF的应答率为50% - 70%,获得性AA和MDS的应答率为40-50%,而骨髓纤维化的应答率非常有限。在获得性AA中,反应与存在非严重疾病相关;在MDS中,雄激素对血小板减少症或轻度至中度贫血更有效,而对输血依赖性贫血的疗效有限。毒性分析主要包括病毒化和肝酶升高,而白血病演变的风险仍存在争议。
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来源期刊
Blood Reviews
Blood Reviews 医学-血液学
CiteScore
13.80
自引率
1.40%
发文量
78
期刊介绍: Blood Reviews, a highly regarded international journal, serves as a vital information hub, offering comprehensive evaluations of clinical practices and research insights from esteemed experts. Specially commissioned, peer-reviewed articles authored by leading researchers and practitioners ensure extensive global coverage across all sub-specialties of hematology.
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